罕见出血性疾病药物研发现状与思考

唐凌; 邹丽敏; 杜瑜; 齐玥丽; 相美亦; 杨志敏

doi:10.12376/j.issn.2097-0501.2022.04.017

罕见出血性疾病药物研发现状与思考

doi: 10.12376/j.issn.2097-0501.2022.04.017

国家药品监督管理局药品审评中心，北京 100022

详细信息

通信作者:
杨志敏, E-mail：yangzhm@cde.org.cn

中图分类号: R554;R96;R973
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- 文章访问数: 91
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- 被引次数: 0
出版历程
- 收稿日期: 2022-08-10
- 录用日期: 2022-09-03
- 网络出版日期: 2022-12-23

Current Development Status and Consideration for Rare Hemorrhagic Disease Drugs

Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China

More Information

Corresponding author: Zhimin, E-mail: yangzhm@cde.org.cn

摘要

摘要: 《第一批罕见病目录》中纳入的罕见遗传性出血性疾病仅血友病一种。然而临床中已知的罕见出血性疾病远不止血友病。与血友病相比，其他大多数罕见出血性疾病更缺乏有效性的治疗手段。血友病是罕见出血性疾病中药物研发较多、较成功的案例，并逐步实现了临床研究与药物研发间的良性循环。药物研发单位可以参考血友病领域的药物研发历程，借鉴血友病药物研发的经验，在罕见出血性疾病中拓宽药物研发领域；通过加强基础研究，实现药物机制创新；借鉴其他罕见病药物研发思路，关注罕见病自然病史研究的重要价值，关注定量药理学工具的应用，提高罕见出血性疾病药物研发效率，并最终满足罕见出血性疾病患者亟待满足的治疗需求。
- 罕见病 /
- 出血性疾病 /
- 药物研发
Abstract: Hemophilia is the only rare hereditary hemorrhagic disorder included in the First Rare Diseases catalogue. However, rare bleeding diseases identified in the clinic are far more common than hemophilia. Most other rare hemorrhagic disorders have less effective treatment than hemophilia. Hemophilia has a history of successful drug development in rare hemorrhagic diseases, and the cycle between clinical research and drug development has been gradually realized. Drug research and pharmaceutical companies can refer to the drug research and development process in the field of hemophilia, learn from the experience of hemophilia drug research and develop treatments. The industry can increase drug development by strengthening basic research, focusing on the value of natural history research, the application of quantitative pharmacological tools and improving the efficiency of drug development to meet the urgent unmet medical needs of patients with rare hemorrhagic diseases.
- rare diseases /
- hemorrhagic diseases /
- drug research and development
注释:

作者贡献： 唐凌负责文章撰写与数据收集；邹丽敏，杜瑜，齐玥丽，相美亦负责收集整理数据；杨志敏负责提出文章构思，并对文章内容进行指导和修改。

利益冲突： 所有作者均声明不存在利益冲突。

HTML全文

图 1 中国血友病药物临床试验分布情况

Figure 1. Distribution of clinical trials of hemophilia drugs in China

下载: 全尺寸图片幻灯片

参考文献(22)

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